Ecopipam, an investigational selective D1 receptor antagonist, previously demonstrated efficacy for the treatment of tics in adults with Tourette syndrome in a phase 2 clinical trial (ClinicalTrials.gov: NCT01244633). In the current phase 2 study (ClinicalTrials.gov: NCT02102698), a team of investigators led by Donald Gilbert, MD, MS, FAAN, FAAP, of Cincinnati Children's Hospital in Ohio, sought to investigate the safety and efficacy of ecopipam in patients aged 7 to 17 years.

The double-blind, placebo-controlled, crossover study included 40 patients with a Yale Global Tic Severity Scale (YGTSS) total tic score of 20. Patients enrolled in the study were not allowed to take ADHD-related stimulants or D2 receptor blocking agents, alpha2 noradrenergic agonists, or other medications for tics. Patients were randomly assigned to receive either ecopipam (50 mg/d for weight <34 kg; 100 mg/d for weight >34 kg) or placebo for 4 weeks, followed by a 2-week washout period and 4-week crossover.

At the 2-week and 4-week evaluations, reductions in total YGTSS scores were greater in the treatment group compared with placebo (P ≤.05). Similar reduction was seen for motor and phonic tics (motor, 16 and 30 days: P ≤.05; phonic, 16 days: P ≤.05 and 30 days: P ≤.09). At 4 weeks, 38% of patients in the treatment group showed “much” or “very much” improvement compared with 18% in the placebo group (P =.08). The proportion of patients whose symptoms were in the “marked” or “severe” range dropped from 40% to 15% after treatment, and the proportion of patients with “mild” symptoms increased from 2.5% to 45%.

Overall, adverse events were mainly mild to moderate, including somnolence, gastrointestinal symptoms, nasopharyngitis, and headache. Four incidents were rated as severe, including headache and insomnia, and 1 was unrelated to treatment. Notably, patients in the treatment group experienced neither weight gain, drug-induced movement disorders, nor changes in renal or hepatic values, electrocardiograms, or vital signs.

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